INDUCED PLURIPOTENT STEM CELLS (IPS CELLS) FOR TREATING LUPUS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 目的：从SLE患者中产生iPS细胞，并将其用作移植用自体原始造血细胞的可扩展来源，以及用于狼疮患者免疫治疗的调节性树突状细胞（DC）和T细胞。 诱导多能干细胞（iPS）是通过插入对维持人胚胎干细胞（hES）的多能性至关重要的某些基因而从体细胞成纤维细胞人工衍生的干细胞。已经表明，这些细胞的行为类似于hES细胞，即能够自我更新和大规模扩增以及向所有三个胚层分化。我们的想法是，iPS细胞可以从SLE患者中产生，并用作移植用自体原始造血细胞的可扩展来源，以及用于免疫治疗的调节性树突状细胞（DC）和T细胞。由于iPS细胞与hES细胞相似，它们可以以高度受控的方式可持续地扩增和遗传改变，这对于更定型的HSC来说是不可能的。因此，iPS细胞可以提供优于体细胞HSC的重要优势，作为基于干细胞的SLE治疗的靶细胞。在本申请中，我们旨在鉴定从iPS细胞产生可移植造血细胞、T细胞和DC所必需的细胞亚群和条件。 这项研究使用了WNPRC干细胞资源。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Objective: To generate iPS cells from SLE patients and use them as a scalable source of autologous primitive hematopoietic cells for transplantation, as well as regulatory dendritic (DC) and T cells for immunotherapy in Lupus patients. Induced pluripotent stem (iPS) cells are stem cells artificially derived from somatic fibroblasts through insertion of certain genes critical for the maintenance of pluripotency of human embryonic stem (hES) cells. It has been shown that these cells behave similar to hES cells, i.e. capable of self-renewal and large-scale expansion and differentiation toward all three germ layers. Our idea is that iPS cells can be generated from SLE patients and used as a scalable source of autologous primitive hematopoietic cells for transplantation, as well as regulatory dendritic (DC) and T cells for immunotherapy. Because iPS cells are similar to hES cells, they can be sustainably expanded and genetically altered in a highly controlled way that is not yet possible with more committed HSCs. Thus, iPS cells could provide important advantages over somatic HSCs as target cells for stem cell based therapy of SLE. In this application we are aiming to identify cell subpopulations and conditions essential for generation of engraftable hematopoietic cells, T cells and DC from iPS cells. This research used WNPRC Stem Cell Resources.